Therapeutic treatment of chromatinopathies

Abstract

Methods for inhibiting Ataxia Telangiectasia and Rad3-related (ATR) protein and Chk1 protein in subjects affected by a chromatinopathy by administering inhibitors of the ATR and Chk1 proteins to the subjects are provided. The chromatinopathy is selected from Kabuki Syndrome (KS), Kabuki Syndrome 2 (KS 2), Charge Syndrome (CS), Rubinstein-Taybi syndrome (RT) and Cornelia de Lange syndrome (CdL).

What is claimed is: 
     
         1 . A method for inhibiting the Ataxia Telangiectasia and Rad3-related (ATR) protein in a subject in need thereof, said method comprising administering to the subject an inhibitor of the ATR protein, wherein said subject is affected by a chromatinopathy. 
     
     
         2 . The method of  claim 1 , wherein the inhibitor of the ATR protein is selected from the group consisting of VX-970, BAY 1895344, AZD6738, AZ20, EPT-46464 and VE-821. 
     
     
         3 . The method of  claim 2 , wherein the inhibitor of the ATR protein is VX-970. 
     
     
         4 . (canceled) 
     
     
         5 . The method of  claim 1 , wherein the chromatinopathy is selected from the group consisting of Kabuki Syndrome (KS), Kabuki Syndrome 2 (KS 2), Charge Syndrome (CS), Rubinstein-Taybi syndrome (RT) and Cornelia de Lange syndrome (CdL). 
     
     
         6 . The method of  claim 5 , wherein the chromatinopathy is Kabuki Syndrome (KS). 
     
     
         7 . A method for inhibiting the Chk1 protein in a subject in need thereof, said method comprising administering to the subject an inhibitor of the Chk1 protein, wherein said subject is affected by a chromatinopathy. 
     
     
         8 . The method of  claim 7 , wherein the inhibitor of the Chk1 protein is selected from the group consisting of GDC-0575, AZD7762, MK-8776, SAR-020106, CCT245737 and PF-477736. 
     
     
         9 . The method of  claim 7 , wherein the chromatinopathy is selected from the group consisting of Kabuki Syndrome (KS), Kabuki Syndrome 2 (KS 2), Charge Syndrome (CS), Rubinstein-Taybi syndrome (RT) and Cornelia de Lange syndrome (CdL). 
     
     
         10 . The method of  claim 9 , wherein the chromatinopathy is Kabuki Syndrome (KS).